2014 Fiscal Year Final Research Report
Development of gene repair therapy using autologous cells for primary immunodeficiencies
Project/Area Number |
22249043
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Research Category |
Grant-in-Aid for Scientific Research (A)
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Allocation Type | Single-year Grants |
Section | 一般 |
Research Field |
Pediatrics
|
Research Institution | Kyushu University |
Principal Investigator |
HARA Toshiro 九州大学, 医学(系)研究科(研究院), 教授 (40150445)
|
Co-Investigator(Kenkyū-buntansha) |
TAKADA Hidetoshi 九州大学, 医学研究院, 教授 (70294931)
IHARA Kenji 大分大学, 医学部, 教授 (80294932)
MIYAWAKI Toshio 富山大学, 医学系研究科, 教授 (10143885)
NAKAHATA Tatsutoshi 京都大学, iPS細胞研究所, 教授 (20110744)
NAKATSU Yoshimichi 九州大学, 医学研究院, 准教授 (00207820)
|
Co-Investigator(Renkei-kenkyūsha) |
ISHIKAWA Fumihiko 理化学研究所, 免疫・アレルギー科学総合研究センター・ヒト疾患モデル研究グループ, グループディレクター (30403918)
|
Project Period (FY) |
2010-04-01 – 2015-03-31
|
Keywords | 遺伝子修復治療 |
Outline of Final Research Achievements |
We constructed a HD-Ad.AAV BTK targeting vector that is composed of the genomic sequence containing BTK exons 6-19, TIMM8A and GFP/hygromycin resistant gene. By using HD-Ad.AAV. BTK vector, we found that the targeting in the BTK gene occurred in 4 among 755 hygromycin-resistant BFU-E colonies differentiated from the cord blood CD34+ cells. Importantly, the gene targeting was also observed in CD19+ lymphoid progenitor cells that could be differentiated from the homologous recombinants of CD34+ cells. Our study shows the potential of HD-Ad.AAV. BTK vector for the BTK gene therapy by homologous recombination in hematopoietic stem cells.
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Free Research Field |
小児科学
|